Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents

CRISPR–Cas9 genome editing is used to correct a dominant-negative mutation in a mouse model of inherited deafness, resulting in improvements in cochlear function and hearing. Hindering heritable hearing loss Nearly half of all deafness cases arise from genetic factors, yet there are limited treatmen...

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Bibliographic Details
Published inNature (London) Vol. 553; no. 7687; pp. 217 - 221
Main Authors Gao, Xue, Tao, Yong, Lamas, Veronica, Huang, Mingqian, Yeh, Wei-Hsi, Pan, Bifeng, Hu, Yu-Juan, Hu, Johnny H., Thompson, David B., Shu, Yilai, Li, Yamin, Wang, Hongyang, Yang, Shiming, Xu, Qiaobing, Polley, Daniel B., Liberman, M. Charles, Kong, Wei-Jia, Holt, Jeffrey R., Chen, Zheng-Yi, Liu, David R.
Format Journal Article
LanguageEnglish
Published London Nature Publishing Group UK 11.01.2018
Nature Publishing Group
Subjects
45
45/41
631/1647/1511
631/61/2297
Acoustic startle response
Acoustic Stimulation
Alleles
Animals
Animals, Newborn
Auditory Threshold
Autosomal dominant inheritance
Base Sequence
Brain stem
Care and treatment
Cell Survival
Cochlea
Cochlea - cytology
Cochlea - metabolism
CRISPR
CRISPR-Associated Proteins - administration & dosage
CRISPR-Associated Proteins - metabolism
CRISPR-Associated Proteins - therapeutic use
CRISPR-Cas Systems
Deafness
Deoxyribonucleic acid
Disease Models, Animal
DNA
Ears & hearing
Evoked Potentials, Auditory, Brain Stem
Female
Fibroblasts
Gene Editing - methods
Gene therapy
Genes
Genes, Dominant - genetics
Genetic disorders
Genetic engineering
Genetic factors
Genetic Therapy - methods
Genome editing
Genomes
Hair Cells, Auditory - cytology
Hearing loss
Hearing Loss - genetics
Hearing Loss - physiopathology
Hearing Loss - prevention & control
Humanities and Social Sciences
Humans
letter
Lipids
Liposomes
Male
Membrane Proteins - genetics
Methods
Mice
multidisciplinary
Mutation
Neonates
Proteins
Reflex, Startle
Ribonucleic acid
RNA
Science
Survival
Online AccessGet full text

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Abstract CRISPR–Cas9 genome editing is used to correct a dominant-negative mutation in a mouse model of inherited deafness, resulting in improvements in cochlear function and hearing. Hindering heritable hearing loss Nearly half of all deafness cases arise from genetic factors, yet there are limited treatment options available for inherited hearing loss. David Liu and colleagues develop a genome-editing approach to target a dominantly inherited form of deafness. In a mouse model of human deafness, CRISPR–Cas9 editing can disrupt the mutant allele and reduce hearing loss. The results support the potential utility of protein–RNA complex delivery in post-mitotic cells as a gene-editing strategy for some autosomal-dominant diseases. Although genetic factors contribute to almost half of all cases of deafness, treatment options for genetic deafness are limited 1 , 2 , 3 , 4 , 5 . We developed a genome-editing approach to target a dominantly inherited form of genetic deafness. Here we show that cationic lipid-mediated in vivo delivery of Cas9–guide RNA complexes can ameliorate hearing loss in a mouse model of human genetic deafness. We designed and validated, both in vitro and in primary fibroblasts, genome editing agents that preferentially disrupt the dominant deafness-associated allele in the Tmc1 (transmembrane channel-like gene family 1) Beethoven ( Bth ) mouse model, even though the mutant Tmc1 Bth allele differs from the wild-type allele at only a single base pair. Injection of Cas9–guide RNA–lipid complexes targeting the Tmc1 Bth allele into the cochlea of neonatal Tmc1 Bth /+ mice substantially reduced progressive hearing loss. We observed higher hair cell survival rates and lower auditory brainstem response thresholds in injected ears than in uninjected ears or ears injected with control complexes that targeted an unrelated gene. Enhanced acoustic startle responses were observed among injected compared to uninjected Tmc1 Bth /+ mice. These findings suggest that protein–RNA complex delivery of target gene-disrupting agents in vivo is a potential strategy for the treatment of some types of autosomal-dominant hearing loss.
AbstractList Although genetic factors contribute to almost half of all cases of deafness, treatment options for genetic deafness are limited. We developed a genome-editing approach to target a dominantly inherited form of genetic deafness. Here we show that cationic lipid-mediated in vivo delivery of Cas9-guide RNA complexes can ameliorate hearing loss in a mouse model of human genetic deafness. We designed and validated, both in vitro and in primary fibroblasts, genome editing agents that preferentially disrupt the dominant deafness-associated allele in the Tmc1 (transmembrane channel-like gene family 1) Beethoven (Bth) mouse model, even though the mutant Tmc1 allele differs from the wild-type allele at only a single base pair. Injection of Cas9-guide RNA-lipid complexes targeting the Tmc1 allele into the cochlea of neonatal Tmc1 mice substantially reduced progressive hearing loss. We observed higher hair cell survival rates and lower auditory brainstem response thresholds in injected ears than in uninjected ears or ears injected with control complexes that targeted an unrelated gene. Enhanced acoustic startle responses were observed among injected compared to uninjected Tmc1 mice. These findings suggest that protein-RNA complex delivery of target gene-disrupting agents in vivo is a potential strategy for the treatment of some types of autosomal-dominant hearing loss.
Although genetic factors contribute to almost half of all cases of deafness, treatment options for genetic deafness are limited1-5. We developed a genome-editing approach to target a dominantly inherited form of genetic deafness. Here we show that cationic lipid-mediated in vivo delivery of Cas9-guide RNA complexes can ameliorate hearing loss in a mouse model of human genetic deafness. We designed and validated, both in vitro and in primary fibroblasts, genome editing agents that preferentially disrupt the dominant deafness-associated allele in the Tmcl (transmembrane channel-like gene family 1) Beethoven (Bth) mouse model, even though the mutant Tmc1Bth allele differs from the wild-type allele at only a single base pair. Injection of Cas9-guide RNA-lipid complexes targeting the Tmc1Bth allele into the cochlea of neonatal Tmc1Bth/+ mice substantially reduced progressive hearing loss. We observed higher hair cell survival rates and lower auditory brainstem response thresholds in injected ears than in uninjected ears or ears injected with control complexes that targeted an unrelated gene. Enhanced acoustic startle responses were observed among injected compared to uninjected Tmc1Bth/+ mice. These findings suggest that protein-RNA complex delivery of target gene-disrupting agents in vivo is a potential strategy for the treatment of some types of autosomal-dominant hearing loss.
Although genetic factors contribute to almost half of all cases of deafness, treatment options for genetic deafness are limited. We developed a genome-editing approach to target a dominantly inherited form of genetic deafness. Here we show that cationic lipid-mediated in vivo delivery of Cas9-guide RNA complexes can ameliorate hearing loss in a mouse model of human genetic deafness. We designed and validated, both in vitro and in primary fibroblasts, genome editing agents that preferentially disrupt the dominant deafness-associated allele in the Tmc1 (transmembrane channel-like gene family 1) Beethoven (Bth) mouse model, even though the mutant Tmc1Bth allele differs from the wild-type allele at only a single base pair. Injection of Cas9-guide RNA-lipid complexes targeting the Tmc1Bth allele into the cochlea of neonatal Tmc1Bth/+ mice substantially reduced progressive hearing loss. We observed higher hair cell survival rates and lower auditory brainstem response thresholds in injected ears than in uninjected ears or ears injected with control complexes that targeted an unrelated gene. Enhanced acoustic startle responses were observed among injected compared to uninjected Tmc1Bth/+ mice. These findings suggest that protein-RNA complex delivery of target gene-disrupting agents in vivo is a potential strategy for the treatment of some types of autosomal-dominant hearing loss.Although genetic factors contribute to almost half of all cases of deafness, treatment options for genetic deafness are limited. We developed a genome-editing approach to target a dominantly inherited form of genetic deafness. Here we show that cationic lipid-mediated in vivo delivery of Cas9-guide RNA complexes can ameliorate hearing loss in a mouse model of human genetic deafness. We designed and validated, both in vitro and in primary fibroblasts, genome editing agents that preferentially disrupt the dominant deafness-associated allele in the Tmc1 (transmembrane channel-like gene family 1) Beethoven (Bth) mouse model, even though the mutant Tmc1Bth allele differs from the wild-type allele at only a single base pair. Injection of Cas9-guide RNA-lipid complexes targeting the Tmc1Bth allele into the cochlea of neonatal Tmc1Bth/+ mice substantially reduced progressive hearing loss. We observed higher hair cell survival rates and lower auditory brainstem response thresholds in injected ears than in uninjected ears or ears injected with control complexes that targeted an unrelated gene. Enhanced acoustic startle responses were observed among injected compared to uninjected Tmc1Bth/+ mice. These findings suggest that protein-RNA complex delivery of target gene-disrupting agents in vivo is a potential strategy for the treatment of some types of autosomal-dominant hearing loss.
Although genetic factors contribute to almost half of all deafness cases, treatment options for genetic deafness are limited 1 – 5 . We developed a genome editing approach to target a dominantly inherited form of genetic deafness. Here we show that cationic lipid-mediated in vivo delivery of Cas9:guide RNA complexes can ameliorate hearing loss in a mouse model of human genetic deafness. We designed and validated in vitro and in primary fibroblasts genome editing agents that preferentially disrupt the dominant deafness-associated allele in the Tmc1 (transmembrane channel-like 1) Beethoven ( Bth ) mouse model, even though the mutant Bth allele differs from the wild-type allele at only a single base pair. Injection of Cas9:guide RNA:lipid complexes targeting the Bth allele into the cochlea of neonatal Bth/+ mice substantially reduced progressive hearing loss. We observed higher hair cell survival rates and lower auditory brainstem response (ABR) thresholds in injected ears compared with uninjected ears or ears injected with complexes that target an unrelated gene. Enhanced acoustic reflex responses were observed among injected compared to uninjected Bth/+ animals. These findings suggest protein:RNA complex delivery of target gene-disrupting agents in vivo as a potential strategy for the treatment of some autosomal dominant hearing loss diseases.
CRISPR–Cas9 genome editing is used to correct a dominant-negative mutation in a mouse model of inherited deafness, resulting in improvements in cochlear function and hearing. Hindering heritable hearing loss Nearly half of all deafness cases arise from genetic factors, yet there are limited treatment options available for inherited hearing loss. David Liu and colleagues develop a genome-editing approach to target a dominantly inherited form of deafness. In a mouse model of human deafness, CRISPR–Cas9 editing can disrupt the mutant allele and reduce hearing loss. The results support the potential utility of protein–RNA complex delivery in post-mitotic cells as a gene-editing strategy for some autosomal-dominant diseases. Although genetic factors contribute to almost half of all cases of deafness, treatment options for genetic deafness are limited 1 , 2 , 3 , 4 , 5 . We developed a genome-editing approach to target a dominantly inherited form of genetic deafness. Here we show that cationic lipid-mediated in vivo delivery of Cas9–guide RNA complexes can ameliorate hearing loss in a mouse model of human genetic deafness. We designed and validated, both in vitro and in primary fibroblasts, genome editing agents that preferentially disrupt the dominant deafness-associated allele in the Tmc1 (transmembrane channel-like gene family 1) Beethoven ( Bth ) mouse model, even though the mutant Tmc1 Bth allele differs from the wild-type allele at only a single base pair. Injection of Cas9–guide RNA–lipid complexes targeting the Tmc1 Bth allele into the cochlea of neonatal Tmc1 Bth /+ mice substantially reduced progressive hearing loss. We observed higher hair cell survival rates and lower auditory brainstem response thresholds in injected ears than in uninjected ears or ears injected with control complexes that targeted an unrelated gene. Enhanced acoustic startle responses were observed among injected compared to uninjected Tmc1 Bth /+ mice. These findings suggest that protein–RNA complex delivery of target gene-disrupting agents in vivo is a potential strategy for the treatment of some types of autosomal-dominant hearing loss.
Audience Academic
Author Lamas, Veronica
Holt, Jeffrey R.
Huang, Mingqian
Thompson, David B.
Kong, Wei-Jia
Liu, David R.
Gao, Xue
Yeh, Wei-Hsi
Liberman, M. Charles
Polley, Daniel B.
Hu, Yu-Juan
Li, Yamin
Wang, Hongyang
Pan, Bifeng
Xu, Qiaobing
Chen, Zheng-Yi
Tao, Yong
Yang, Shiming
Hu, Johnny H.
Shu, Yilai
AuthorAffiliation 5 Department of Otorhinolaryngology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, Hubei 430022, China
4 Department of Otolaryngology and Program in Neuroscience, Harvard Medical School and Eaton Peabody Laboratory, Massachusetts Eye and Ear Infirmary, Boston, Massachusetts 02114, USA
9 Department of Biomedical Engineering, Tufts University, Medford, MA 02155, USA
7 Departments of Otolaryngology and Neurology, F.M. Kirby Neurobiology Center Boston Children’s Hospital, and Harvard Medical School, Boston, Massachusetts 02115, USA
8 Department of Otolaryngology–Head and Neck Surgery, Eye and ENT Hospital, Shanghai Medical College, Fudan University, Shanghai, China
3 Broad Institute of MIT and Harvard, Cambridge, MA, 02141, USA
6 Program in Speech and Hearing Bioscience and Technology, Harvard University, Cambridge, Massachusetts 02138, USA
2 Howard Hughes Medical Institute, Harvard University, Cambridge, MA, 02138, USA
10 Department of Otolaryngolog
AuthorAffiliation_xml – name: 1 Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA, 02138, USA
– name: 3 Broad Institute of MIT and Harvard, Cambridge, MA, 02141, USA
– name: 2 Howard Hughes Medical Institute, Harvard University, Cambridge, MA, 02138, USA
– name: 4 Department of Otolaryngology and Program in Neuroscience, Harvard Medical School and Eaton Peabody Laboratory, Massachusetts Eye and Ear Infirmary, Boston, Massachusetts 02114, USA
– name: 10 Department of Otolaryngology & Head Neck Surgery, Key Lab of Hearing Impairment Science of Ministry of Education, Key Lab of Hearing Impairment Prevention and Treatment of Beijing City, Chinese PLA Medical School, Beijing, China
– name: 6 Program in Speech and Hearing Bioscience and Technology, Harvard University, Cambridge, Massachusetts 02138, USA
– name: 9 Department of Biomedical Engineering, Tufts University, Medford, MA 02155, USA
– name: 5 Department of Otorhinolaryngology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, Hubei 430022, China
– name: 7 Departments of Otolaryngology and Neurology, F.M. Kirby Neurobiology Center Boston Children’s Hospital, and Harvard Medical School, Boston, Massachusetts 02115, USA
– name: 8 Department of Otolaryngology–Head and Neck Surgery, Eye and ENT Hospital, Shanghai Medical College, Fudan University, Shanghai, China
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  surname: Gao
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  givenname: Yong
  surname: Tao
  fullname: Tao, Yong
  organization: Department of Otolaryngology and Program in Neuroscience, Harvard Medical School and Eaton Peabody Laboratory, Massachusetts Eye and Ear Infirmary, Department of Otorhinolaryngology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Department of Otolaryngology-Head and Neck Surgery, Shanghai Ninth People’s Hospital and Ear Institute, Shanghai Jiaotong University School of Medicine
– sequence: 3
  givenname: Veronica
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  givenname: Mingqian
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  surname: Yeh
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– sequence: 6
  givenname: Bifeng
  surname: Pan
  fullname: Pan, Bifeng
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  givenname: Yu-Juan
  surname: Hu
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– sequence: 8
  givenname: Johnny H.
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  organization: Department of Chemistry and Chemical Biology, Harvard University, Howard Hughes Medical Institute, Harvard University
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  surname: Shu
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– sequence: 11
  givenname: Yamin
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  fullname: Wang, Hongyang
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  surname: Kong
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BackLink https://www.ncbi.nlm.nih.gov/pubmed/29258297$$D View this record in MEDLINE/PubMed
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Current address: Department of Chemical and Biomolecular Engineering, Rice University, Houston, Texas 77005, USA
These authors contributed equally to this work
Current address: Department of Otolaryngology-Head and Neck Surgery, Shanghai Ninth People’s Hospital and Ear Institute, Shanghai Jiaotong University School of Medicine, Shanghai, 200011, China
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  publication-title: J. Assoc. Res. Otolaryngol.
  doi: 10.1007/s10162-017-0634-8
– volume: 33
  start-page: 73
  year: 2015
  ident: BFnature25164_CR15
  publication-title: Nat. Biotechnol.
  doi: 10.1038/nbt.3081
– reference: 29323325 - Nature. 2018 Jan 11;553(7687):162-163. doi: 10.1038/d41586-017-08645-z.
– reference: 29293216 - Nature. 2017 Dec 21;552(7685):300-301. doi: 10.1038/d41586-017-08722-3.
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Snippet CRISPR–Cas9 genome editing is used to correct a dominant-negative mutation in a mouse model of inherited deafness, resulting in improvements in cochlear...
Although genetic factors contribute to almost half of all cases of deafness, treatment options for genetic deafness are limited. We developed a genome-editing...
Although genetic factors contribute to almost half of all cases of deafness, treatment options for genetic deafness are limited1-5. We developed a...
Although genetic factors contribute to almost half of all deafness cases, treatment options for genetic deafness are limited 1 – 5 . We developed a genome...
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StartPage 217
SubjectTerms 45
45/41
631/1647/1511
631/61/2297
Acoustic startle response
Acoustic Stimulation
Alleles
Animals
Animals, Newborn
Auditory Threshold
Autosomal dominant inheritance
Base Sequence
Brain stem
Care and treatment
Cell Survival
Cochlea
Cochlea - cytology
Cochlea - metabolism
CRISPR
CRISPR-Associated Proteins - administration & dosage
CRISPR-Associated Proteins - metabolism
CRISPR-Associated Proteins - therapeutic use
CRISPR-Cas Systems
Deafness
Deoxyribonucleic acid
Disease Models, Animal
DNA
Ears & hearing
Evoked Potentials, Auditory, Brain Stem
Female
Fibroblasts
Gene Editing - methods
Gene therapy
Genes
Genes, Dominant - genetics
Genetic disorders
Genetic engineering
Genetic factors
Genetic Therapy - methods
Genome editing
Genomes
Hair Cells, Auditory - cytology
Hearing loss
Hearing Loss - genetics
Hearing Loss - physiopathology
Hearing Loss - prevention & control
Humanities and Social Sciences
Humans
letter
Lipids
Liposomes
Male
Membrane Proteins - genetics
Methods
Mice
multidisciplinary
Mutation
Neonates
Proteins
Reflex, Startle
Ribonucleic acid
RNA
Science
Survival
Title Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents
URI https://link.springer.com/article/10.1038/nature25164
https://www.ncbi.nlm.nih.gov/pubmed/29258297
https://www.proquest.com/docview/1987371515
https://www.proquest.com/docview/1979171552
https://pubmed.ncbi.nlm.nih.gov/PMC5784267
Volume 553
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